Shots:
The US FDA has approved Ctexli (chenodiol) to treat cerebrotendinous xanthomatosis (CTX) in adults
Efficacy was established in crossover withdrawal P-III (RESTORE) trial assessing Ctexli (250mg, PO, TID) vs PBO in pts for 24wks.
Study showed reduced levels of plasma cholestanol & urine 23S-pentol in pts compared to PBO
Ref: Prnewswire | Image: Mirum
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